Second Generation Mitochondrial Targeted Candidate Enters Clinic with Potential to Treat Neurodegenerative Diseases
BOSTON, Jan. 21, 2020 /PRNewswire/ -- Stealth BioTherapeutics (NASDAQ: MITO), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced the initiation of a first-in-human Phase 1 trial evaluating its second generation pipeline compound, SBT-272, in healthy subjects.
"We are excited to advance our second generation of mitochondrial therapeutics into the clinic," said Reenie McCarthy, Chief Executive Officer of Stealth. "We designed this product candidate for neurodegenerative diseases, aiming to improve potency and blood-brain barrier penetration, and we're encouraged by early signals of its efficacy in a preclinical study in amyotrophic lateral sclerosis (ALS). Importantly, our preclinical work identified a responsive biomarker, which may help inform our ongoing preclinical studies in ALS and multiple system atrophy (MSA), as well as future development efforts in other neurodegenerative diseases in which mitochondrial dysfunction has been implicated."
The Phase 1 trial is a double-blind, placebo-controlled, single-ascending dose study enrolling up to 40 healthy subjects across multiple cohorts. SBT-272 is being administered orally in the study. As a primary objective, the study will evaluate safety and tolerability of SBT-272. Secondary objectives include an analysis of the pharmacokinetic profile and appropriate dose range.
SBT-272 is a novel peptidomimetic being developed for the treatment of neurodegenerative diseases involving mitochondrial dysfunction. SBT-272 has been shown to increase adenosine triphosphate (ATP) production and decrease levels of reactive oxygen species (ROS) in dysfunctional mitochondria in preclinical studies. SBT-272 demonstrates higher mitochondrial uptake, greater concentrations in the brain, and improved oral bioavailability relative to elamipretide, Stealth's first-in-class lead compound. Treatment with SBT-272 was associated with a dose-dependent delay in the onset of neurological disease, a reduction in systemic markers of neurodegeneration and prolonged lifespan in a mouse model of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease characterized by motor neuron deterioration and muscle atrophy. The compound is currently being evaluated in another ALS preclinical model, as well as in a preclinical model indicative of activity in multiple system atrophy (MSA), a neurological disorder leading to parkinsonism, cerebellar ataxia, dysautonomia and other motor and non-motor symptoms. Mitochondrial dysfunction is believed to contribute to the progression of ALS and MSA, as well as other neurodegenerative diseases including Parkinson's, Huntington's and Alzheimer's.
We are a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction. Mitochondria, found in nearly every cell in the body, are the body's main source of energy production and are critical for normal organ function. Dysfunctional mitochondria characterize a number of rare genetic diseases, collectively known as primary mitochondrial diseases, and are also involved in many common age-related diseases. We believe our lead product candidate, elamipretide, has the potential to treat both rare genetic and common age-related mitochondrial diseases. We are studying elamipretide in Barth syndrome, geographic atrophy associated with dry age-related macular degeneration and Leber's hereditary optic neuropathy. Our newest clinical stage candidate, SBT-272, is being evaluated for rare neurodegenerative disease indications. Our pipeline compounds include SBT-259, which we are evaluating for rare peripheral neuropathies, and the SBT-550 series of compounds, which we plan to evaluate for rare neurodegenerative and ophthalmic disease indications. We have optimized our discovery platform to identify novel mitochondria-targeted compounds, which may be nominated as therapeutic product candidates or utilized as scaffolds to deliver other compounds to mitochondria. We have assembled a highly experienced management team, board of directors and group of scientific advisors to help us achieve our mission of leading mitochondrial medicine.
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the ongoing Phase 1 clinical trial of SBT-272, the potential of SBT-272 to target mitochondrial dysfunction, the potential of SBT-272 as a treatment for neurodegenerative diseases such as ALS and MSA, and Stealth's plans, strategies and expectations for its preclinical and clinical advancement of SBT-272 and its other pipeline candidates. Statements that are not historical facts, including statements about Stealth's beliefs, plans and expectations, are forward-looking statements. The words "anticipate," "expect," "hope," "plan," "potential," "possible," "will," "believe," "estimate," "intend," "may," "predict," "project," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Stealth may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of known and unknown risks, uncertainties and other important factors, including: Stealth's ability to obtain additional funding; the ability to successfully demonstrate the efficacy and safety of Stealth's product candidates and future product candidates; the preclinical and clinical results for Stealth's product candidates, which may not support further development and marketing approval; the potential advantages of Stealth's product candidates; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies, which may affect the initiation, timing and progress of preclinical studies and clinical trials of Stealth product candidates; Stealth's ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Stealth's ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Stealth's most recent Annual Report on Form 20-F filed with the Securities and Exchange Commission ("SEC"), as well as in any future filings with the SEC. Forward-looking statements represent management's current expectations and are inherently uncertain. Except as required by law, Stealth does not undertake any obligation to update forward-looking statements to reflect subsequent events or circumstances.
Stern Investor Relations
Lauren Stival, 212-362-1200
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